Advancements in leukemia treatment for children are a success story that has laid the groundwork for other cancer treatments for children.

Each year, 15 to 20 children in Uusimaa are diagnosed with the most common kind of pediatric cancer, acute lymphoblastic leukemia (ALL). The treatment for the condition lasts about two years. Treatment results at the New Children’s Hospital are of top quality. Of the patients in our care, depending on the risk classification of their leukemia, up to 98% are cured. This success is the result of treatment programs based on cytokine combination therapy and close international collaboration.

However, not all leukemia patients can be cured with cytokine therapy. New smart drugs or treatments are needed in addition. The most recent addition to the palette of ALL treatments is CAR-T-cell therapy (Chimeric Antigen Receptor T-cell therapy). The New Children’s Hospital is the only one in Finland to administer CAR-T-cell therapy to children, and as such offers this treatment to patients from anywhere in the country.

“CAR-T-cell therapy is an alternative leukemia treatment when conventional cytokine treatment elicits no response or if the leukemia recurs. For some patients, leukemia can recur even after CAR-T-cell therapy, but in about 80% of these patients with the most difficult conditions the CAR-T-cell therapy produces a response. We are experimentally extending this treatment to lymphoma patients. Ten patients have received CAR-T-cell therapy since 2019, and our treatment results are excellent,” says Chief Physician Mervi Taskinen.

“We entered into negotiations with the pharmaceutical company as soon as the European Medicines Agency (EMA) granted a marketing authorization for the medication in 2018, and towards the end of 2018 we were approved as a provider of CAR-T-cell therapy as the first center in the Nordic countries. We are involved in two research projects the purpose of which is to establish CAR-T-cell therapy as the front line treatment for childhood ALL in the patients with the worst prognoses and also for cases of recurring or non-responsive childhood lymphoma. My research group is currently working on the next big ‘tool’ in smart cell therapy, known as Natural Killer (NK) cells,” says Kim Vettenranta, Professor of Cell Therapy and Transfusion Medicine at the University of Helsinki.

What are CAR-T-cells?

CAR-T-cells are a customized smart drug manufactured from the patient’s own lymphocytes. These lymphocytes are harvested from the patient’s bloodstream and genetically modified at a laboratory at the pharmaceutical plant so that they are able to identify a structure on the surface of a leukemia cell.

When a CAR-T-cell encounters leukemia cells in the patient’s body, a reaction is initiated that leads to the destruction of the leukemia cell. In order for the treatment to have long-lasting effects, the CAR-T-cells must be able to reproduce and survive in the body. It is not known yet exactly how long the CAR-T-cells have to survive in the body for the condition to be cured.